Welcome to NORD
News and Events
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FDA and EMA to Co-Host Designation Workshop FDA OOPD and its European counterpart will host an Orphan Designation and Grant workshop in Silver Spring, MD, on Oct. 12. Learn more about the meeting > |
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Conference on Clinical Research for Rare Diseases The NIH Rare Diseases Clinical Research Network and the Clinical and Translational Science Awards are sponsoring the 3rd Conference on Clinical Research for Rare Diseases on October 2 in Rockville, MD. Learn More > |
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Christopher Austin to Serve as First NCATS Director NIH Director Francis Collins has announced that Christopher Austin MD will serve as the first director of the National Center for Advancing Translational Sciences. Read more > |
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President Obama has signed into law a bill very important to rare disease patients. Read op-ed piece by NORD's president. > |
News for Patients
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Where States Stand on the Medicaid Expansion The US Supreme Court has ruled that states can effectively opt out of the ACA’s Medicaid expansion without any effect on current funding. American Health Line has compiled information about each state. More > |
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Lysosomal Storage Disorders Survey A student researcher is conducting an interview study of adults with lysosomal storage disorders who have been involved in any clinical study or registry. Access the survey > |
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Regional Teleconference with CMS CMS will be hosting a series of regional teleconferences in September to discuss Health Insurance Marketplace Exchanges and expanded insurance coverage options. More > |
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NBS Connect is a new internet-based support network for parents, guardians and individuals with inborn errors of metabolism. Learn more > |
News for Patient Organizations
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Share Information With NORD About Your Awareness Dates NORD would like to promote awareness and support for disease-specific observances. Submit your information here> |
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NORD Organizational Membership Meeting in Philadelphia Members should register now for NORD’s Regional Membership Meeting in Philadelphia on Friday, September 21st. Learn more > |
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FDA OOPD Workshop on Pediatric Device Development This free workshop will be held at the FDA's White Oak campus in Silver Spring, Maryland on Monday, September 24. Read more and register here > |
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Gene for Alternating Hemiplegia of Childhood Identified One of NORD's member organizations played a key role in identifying the gene for this disease. Read more > |
News for Medical Professionals
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Proposed Legislation Would Bolster Reporting of Clinical Trial Data The TEST Act would require all US biomedical studies on humans be registered on ClinicalTrials.gov and that the results be reported within one year. More> |
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Conference on Clinical Research for Rare Diseases The NIH Rare Diseases Clinical Research Network and the Clinical and Translational Science Awards are sponsoring the 3rd Conference on Clinical Research for Rare Diseases on October 2. More > |
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CRN Develops Resource for Cystinosis Treatment Cystinosis Research Network develops standards of care resource for families and physicians. Read more > |
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NIH Seeks Proposals to Study Genomic Sequencing in Newborn Period The NIH is intending to fund projects to further the understanding of newborn on-set disorders and to improve treatments for these diseases. Read More > |
News for Industry
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NORD Board Members Honored by PharmaVoice Two members of NORD's Board of Directors have been named to the "PharmaVoice 100". Read more > |
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Study Compares Oncology Drug Approvals and Costs Tufts study finds oncology drugs approved more quickly in U.S. than in Europe but, in general, more costly. More > |
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FDA-NIH Science of Small Clinical Trials This course will present an overall framework and provide training in the scientific aspects of designing and analyzing clinical trials based on small study populations. Read more> |
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NORD's President Interviewed about Rare Disease R&D NORD President Peter L. Saltonstall was interviewed recently by PharmaPhoroum. Read the article > |
